Medulloblastoma is the most common malignant pediatric brain tumor consisting of at least four distinct molecular subgroups: Wingless (WNT), sonic hedgehog (SHH), Group 3 and Group 4. These subgroups are characterized by divergent genetic aberrations, cytogenetic features, and distinct phenotypes including patient demographics and clinical outcome. Tumors with WNT pathway activation have the most favorable prognosis whereas Group 3 medulloblastomas have the worst outcome. Group 3 tumors are restricted to pediatric patients, characterized by amplification of MYC (a regulator gene encoding a transcription factor), and are frequently metastatic at the time of diagnosis. These tumors are particularly resistant to conventional therapies with radiation and chemotherapy, even at maximally tolerated doses, highlighting the need for novel and more effective therapeutic options.
It would be desirable, thus, to develop a therapy useful to treat a medulloblastoma.